That Prescription Might Not Have Been Tested For Your Ailment

It's actually quite common for doctors to write "off label" prescriptions, including using cancer drugs to treat migraine headaches or blood pressure medication for heart failure.

One study found that 1 in 5 prescriptions written in doctor's offices has not been approved by the Food and Drug Administration to treat the condition it is being used for. And while some off-label drugs are used with no problems, others may not work or may increase a patient's risk of complications.

There are many reasons why a doctor will write an off-label prescription. It could be a last resort. It could be a cancer drug that shows promise when all other options have failed. Or it could be anecdotal evidence from other doctors who say the medication works for certain conditions. Dr. Caleb Alexander, who co-directs the Johns Hopkins Center for Drug Safety and Effectiveness, says patients may not even know that prescriptions they get are off label. "For example, the use of anti-seizure drugs to treat depression or neuropathic pain, or the use of certain cancer drugs to treat macular degeneration, " he says.

Off-label drug prescribing is neither bad nor good per se, says Dr. Randall Stafford, an epidemiologist with the Stanford Prevention Research Center. There may be studies and research showing the drugs safe and effective to treat that condition. But there may not be. In most cases, Stafford says, the evidence just doesn't exist.

And that can be risky. For example, antipsychotic medications are often used to treat patients with advanced dementia who have problems sleeping at night. The antipsychotics often act as a mood stabilizer and have a sedating effect. But "the medication can increase the risk of heart attack in this elderly population," Stafford says.

So why aren't these medications FDA approved for those uses? It boils down to money.

It can cost drug companies hundreds of millions of dollars to do the research and perform the clinical trials necessary to meet the rigorous demands of FDA approval. They've done that once for the initial approval, and many are not willing to do it again for a secondary use.

Alexander says many companies view it as simply not worth it, especially if there are only a few years left in a drug's patent life. Once the drug goes generic, he says, it's not as profitable.

So that often leaves decisions up to the individual doctor. "Clinicians are between a rock and a hard place, because there are thousands of drugs on the market," Alexander says, "and they face a big challenge knowing all they need to know about all the drugs."

In one survey of doctors, Alexander found that in about half of all cases where medication was prescribed, doctors didn't know whether it was FDA approved for the condition. This is why researchers say it's so important to have a comprehensive nationwide database for doctors to consult when considering medication that's not approved by the FDA for that use.

Until such a database is available, Alexander says, it will be up to patients and their doctors to have frank discussions about what evidence is available and weigh the pros and cons of using certain medications off label.

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Transcript

STEVE INSKEEP, HOST:

Now it's not just patients with hepatitis C who might be prescribed a drug combination that has not been approved by the FDA. It's actually quite common for doctors to prescribe drugs off-label, as it's called. In fact, one of five drugs are prescribed this way.

NPR's Patti Neighmond has been asking why.

PATTI NEIGHMOND, BYLINE: There are many reasons why doctors prescribe drugs off-label - either alone or in combinations. It could be a last resort. A cancer drug that shows promise when all other options have failed. Or it could be anecdotal evidence from other doctors who say the medication works for certain conditions.

Dr. Caleb Alexander co-directs the Johns Hopkins Center for Drug Safety and Effectiveness. He says patients may not know it but their prescriptions are often made for conditions that haven't been scrutinized or approved by the FDA.

DR. CALEB ALEXANDER: For example, the use of anti seizure drugs to treat migraine headaches or depression or neuropathic pain.

NEIGHMOND: Or the use of certain cancer drugs to treat macular degeneration or blood pressure medication to treat heart failure or help prevent headaches, all considered off-label.

NEIGHMOND: Internist Randall Stafford with the Stanford Prevention Research Center.

STAFFORD: But really depends on the level of evidence available to support a particular use of a drug for a condition that may not have been specifically approved by the FDA.

NEIGHMOND: Evidence is the critical word here. For some off-label use research and studies show they're effective. But they're not FDA approved to treat these health problems. Researcher Alexander says that's because drug companies often don't have a financial incentive to spend the hundreds of millions of dollars it could cost to get FDA approval for an additional use.

ALEXANDER: If there's only a few years left in a drug's patent life, then pretty soon the drug will be going generic and pharmaceutical manufacturers face little incentive to seek an additional FDA approval.

NEIGHMOND: So that often leaves decisions up to the individual doctor.

ALEXANDER: Clinicians are between a rock and a hard place because there are thousands of drugs on the market and many clinicians use dozens of medicines at least to treat patients.

NEIGHMOND: In one survey of doctors, Alexander found in about half of all cases where medications were prescribed, doctors didn't know whether they were FDA approved for the specific condition.

Stanford's Randall Stafford says that can result in risky prescribing. For example, some doctors use anti-psychotic medication to treat patients with advanced dementia who have problems sleeping at night.

STAFFORD: They may have reversal of day and night and medications in the class of anti-psychotics often act as a mood stabilizer and have sedating properties.

NEIGHMOND: Which may make it easier to care for these patients but at the same time can increase their risk of heart attack.

Researchers agree it's complications like this that make it so important to have a comprehensive nationwide database for doctors to consult when considering medication that's not approved by the FDA.

Until that's available, it'll be up to patients and their doctors to discuss the pros and cons of using medication off-label.